Safety and Pharmacokinetics of Rezafungin

Study Summary

This is a Phase 1, double-blind, placebo-controlled trial in three parts. A single ascending dose (SAD) study in six cohorts receiving a single subcutaneous (SC) dose of 1, 10, 30, 60, 100, or 200 mg of rezafungin; a multiple ascending dose (MAD) study in four cohorts receiving 30 mg x 3 doses, 60 mg x 3 doses, 100 mg x 3 doses, or 200 mg x 3 doses of rezafungin SC with dosing frequency of once every 7 days; and a two-period cross-over bioavailability (BA) study receiving 100 mg of rezafungin. The two period cross-over BA study will be assessed unblinded in two sequences (10 subjects, 100 mg or maximum tolerated dose (MTD) of rezafungin in Part 1); 5 subjects will receive an SC injection of rezafungin in Period 1 followed by an intravenous (IV) infusion of rezafungin in Period 2, and 5 subjects will receive an IV infusion of rezafungin in Period 1 followed by an SC injection of rezafungin in Period 2. Each SAD (except cohort 1) and MAD cohort will contain 8 subjects (6 subjects will receive a SC injection of rezafungin and 2 subjects will receive placebo). Each SAD (except cohort 1) and MAD cohort will be conducted with sentinel dosing. SAD cohort 1 will be comprised of 4 subjects (3:1 rezafungin to placebo) with no sentinel dosing. Parts 2 and 3 of the study will only be conducted after FDA review for safety data and PK data from all subjects participating in Part 1; Part 3 may be run in parallel with the first cohort (Cohort 7) of Part 2. Individuals in the SAD cohorts will participate for approximately 58 days, including up to 28 days for screening and 30 days for dosing and follow-up (FU). Individuals in the MAD cohorts will participate for approximately 73 days, including up to 28 days for screening and 45 days for dosing and FU. Individuals in the BA cohorts will participate for approximately 80 days, including up to 28 days for screening and 52 days for dosing and FU. The study will have a duration of approximately 30 months. The primary objectives are to det

Primary Outcome

Laboratory parameters and associated thresholds include albumin \<=3.4 g/dL, glucose \<= 69 mg/dL or \>=106 mg/dL, blood urea nitrogen (BUN) \>= 21 mg/dL, potassium \>=5.2 mEq/L or \<=3.4 mEq/L, calcium \< 8.7 mg/dL or \>=10.3 mg/dL, sodium \<=132 mEq/L or \>=144 mEq/L, total protein \<=5.9 g/dL, creatinine \>=1.3 mg/dL (male) or \>= 1.0 mg/dL (female), creatine phosphokinase \>= 309 U/L, phosphorus \<=2.4 mg/dL, alkaline phosphatase \>= 131 IU/L (males) or \>= 106 IU/L (female), aspartate amino

Interventions

• OTHER Placebo
• DRUG Rezafungin